Health Outcomes Associated With Transition From Pediatric to Adult Cystic Fibrosis Care

Division of Adolescent and Young Adult Medicine and Center for Translational Science, Children's National Medical Center, Washington, District of Columbia
PEDIATRICS (Impact Factor: 5.3). 10/2013; 132(5). DOI: 10.1542/peds.2013-1463
Source: PubMed

ABSTRACT BACKGROUND:Almost half of individuals who have cystic fibrosis (CF) are over 18 years old, thus safely transferring patients from pediatric to adult care is a priority. The purpose of this study is to compare youth transferred from pediatric to adult CF care versus those remaining in pediatric CF care and quantify the relationship between transfer status and health outcomes.METHODS:Patients who transferred from pediatric to adult CF care were identified from the CF Foundation Patient Registry from 1997 to 2007. Transferred patients were compared with individuals who have similar baseline characteristics who remained in pediatric care throughout the same time period. The main outcome measures include pulmonary function, nutritional status, care use, and home intravenous antibiotic events per year. A propensity-matched analysis was performed.RESULTS:Fifty-eight percent of patients remained in pediatric programs throughout the study period. The mean age at transfer to adult care was 21.2 (1.3) years. In the 2 years after transfer there was a less rapid decline in percent predicted forced expiratory volume in 1 second of 0.78 percentage points per year among transfer-positive patients compared with transfer-negative ones (95% confidence interval; 0.06-1.51); there were no other significant health related changes.CONCLUSIONS:The current study contradicts reports of other chronic childhood conditions, in which transfer between the pediatric and adult health system was associated with adverse health outcomes. Further research is needed to explore the long-term relationship between transition practices and health status outcomes to establish a systematic, evidence-based transition process.

  • [Show abstract] [Hide abstract]
    ABSTRACT: Introduction: Data on disease burden of cystic fibrosis in Hungary are scarce. Aim: To assess quality of life and resource utilisations of patients with cystic fibrosis. Method: In a cross-sectional survey (BURQOL-RD project), the EQ-5D-5L questionnaire was applied and healthcare utilisations were retrospectively surveyed. Results: 110 patients participated in the study (age-groups, year: 0-13, N = 48; 14-17, N = 12; ≥18, N = 50), median age at the diagnosis was 1 year. EQ-5D-5L score in age-groups 18-24 and 25-34 was significantly lower than in the general population (p<0.05). 75 patients (68%) attended pulmonology care, 55 patients (50%) were hospitalised in the past 6 and 12 months, respectively, and 57 patients (52%) were taking dornase alpha. Five adult patients (10%) received help from non-professional caregiver. Conclusions: Cystic fibrosis leads to significant deterioration of quality of life. This study is the first from the Central Eastern European region that provides basic inputs for further health economic evaluations of cystic fibrosis care. Orv. Hetil., 2014, 155(42), 1673-1684.
    Orvosi Hetilap 10/2014; 155(42):1673-1684. DOI:10.1556/OH.2014.30016
  • [Show abstract] [Hide abstract]
    ABSTRACT: Congenital neuromuscular disorders, such as Duchenne muscular dystrophy (DMD), spinal muscular atrophy (SMA), and Pompe disease (acid maltase deficiency, AMD) are candidates for universal newborn screening (NBS). In this paper, we discuss the future path of NBS for these disorders with particular emphasis on DMD-NBS, because of the likely approval of new gene modifying treatments, the possible benefits of earlier treatment with corticosteroids, and the recently demonstrated feasibility of a two tiered approach to NBS with screening by creatine kinase (CK) levels in dried blood spots followed by mutation detection in those with elevated CK.The cystic fibrosis newborn screening (CF-NBS) program is a successful model for NBS. Cystic fibrosis (CF) outcomes have consistently improved into adulthood following introduction of CF-NBS because considerable resources have been devoted to practices that include: attention to improving laboratory screening; consistent confirmatory testing and immediate referral of all newly diagnosed infants to designated CF care centers that follow established practice guidelines; and, ongoing evaluation of CF care centers via a centralized clinical database.Like CF, DMD, SMA, and infantile AMD are inexorably debilitating and require lifetime multidisciplinary clinical management. NBS would address the delays in diagnosis that prevent patients from receiving timely treatments. Standardized care following early diagnosis would reduce disparities in clinical care and outcomes. NBS in these neuromuscular disorders should be implemented, utilizing lessons learned from the past 20 years of CF-NBS: standardized protocols for all patients identified by DMD-NBS; longitudinal follow-up in multidisciplinary clinics; and coordinated oversight of these clinics. This article is protected by copyright. All rights reserved.
    Annals of Neurology 11/2014; 77(2). DOI:10.1002/ana.24316 · 11.91 Impact Factor
  • [Show abstract] [Hide abstract]
    ABSTRACT: We previously reported that indirect and intangible costs burden households with a food allergic adult. We now extend our investigation to households with food allergic children and adolescents. The objective of this study was to estimate direct, indirect, and intangible costs of food allergy in households with a child and/or adolescent with objectively diagnosed allergy to staple foods (cow's milk, hen's egg, and/or wheat), and to compare these costs with age- and sex-matched controls. Direct and indirect cost parent-reported data collected via the Food Allergy Socio-Economic Questionnaire of 84 children (0-12 years) and 60 adolescents (13-17 years) with objectively diagnosed allergy to staple foods ("cases") and age- and sex-matched controls (n = 94 children; n = 56 adolescents) were compared. Annual household costs were calculated. Total household costs included direct plus indirect costs. Intangible costs included parent-reported health of their child and/or adolescent, standard of living, and perceptions of well-being. Amongst cases, total household costs were higher by €3961 for children and €4792 for adolescents versus controls (P < .05), and were driven by direct (eg, medications) and indirect (eg, time with health care professionals) costs. For children only, a history of anaphylaxis was associated with higher direct costs than no anaphylaxis (€13,016 vs €10,044, P < .05). Intangible costs (eg, parent-reported health of a child and/or adolescent) were significantly impacted amongst cases versus controls (P < .01). Households with a child and/or adolescent with objectively diagnosed allergy to staple foods have higher total household costs than controls. Direct and indirect costs were significantly higher for cases versus controls amongst children only. Amongst both age groups, such allergy adversely impacted intangible costs. Copyright © 2015 The Authors. Published by Elsevier Inc. All rights reserved.